FDA Memberikan Penunjukan Penyakit Langka Pediatric untuk SAT-3247 untuk Mengobati Distrofi Otot Duchenne

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Referensi

1. Satellos Receives Rare Pediatric Disease Designation from the U.S. FDA for SAT-3247 for the Treatment of Duchenne Muscular Dystrophy. Satellos. News release. August 8, 2024. Accessed August 9, 2024. https://www.businesswire.com/news/home/20240808320005/en/Satellos-Receives-Rare-Pediatric-Disease-Designation-from-the-U.S.-FDA-for-SAT-3247-for-the-Treatment-of-Duchenne-Muscular-Dystrophy

2. Duchenne Muscular Dystrophy. National Library of Medicine. News release. July 10, 2023. Accessed August 9, 2024. https://www.ncbi.nlm.nih.gov/books/NBK482346/#:~:text=Duchenne%20muscular%20dystrophy%20(DMD)%20is%20one%20of%20the%20most%20severe,muscle%20fiber%20degeneration%20and%20weakness.

3. Lambert P. LB: SAT-3247: An Oral Small Molecule Inhibitor Targeting AAK1, a Critical Effector Of Skeletal Muscle Regeneration. MDA Clinical & Scientific Conferece. March 2024. Accessed August 9, 2024. https://www.mdaconference.org/abstract-library/sat-3247-an-oral-small-molecule-inhibitor-targeting-aak1-a-critical-effector-of-skeletal-muscle-regeneration/

4. Satellos Announces Submission of Regulatory Filing to Commence a Phase 1 Clinical Trial with SAT-3247. Satellos. News release. July 11, 2024. Accessed August 9, 2024. https://ir.satellos.com/news/news-details/2024/Satellos-Announces-Submission-of-Regulatory-Filing-to-Commence-a-Phase-1-Clinical-Trial-with-SAT-3247/default.aspx